Alnylam Blames New US Drug Pricing Laws for Shelving of Rare Eye Disease Therapeutic
Alnylam is setting aside a clinical trial of its therapeutic Vutrisiran for a rare eye disease due to the looming US drug pricing reforms, according to the company. The RNA-based treatment for Stargardt disease is currently used in another rare disease but would not face Medicare pricing negotiations because the law only targets newly approved drugs. Approval of the treatment for Stargardt would allow the government to negotiate the price of Vutrisiran for that indication.
AJMC “What We’re Reading” – Uninsured Children, HIV and Monkeypox, and Health Outcomes by US State
In the latest edition of “What We’re Reading,” the AJMC staff cover several breaking stories. A recent study found that people living in conservative US states have lower life expectancies than more liberal ones. The researchers’ anaysis shows that over 170,000 lives could be saved if certain liberal policies were enacted nationwide. In other news, new data shows a striking comorbidity between monkeypox and HIV.
HIV/AIDS Patient Advocacy Organization Incensed by Gilead’s 340B Descovy Price Hikes
Gilead Sciences’ refusal to provide Medicare 340B discounts for HIV drugs has drawn significant ire from HIV/AIDS patient advocacy organizations. The AIDS Healthcare Foundation (AHF) said that the pharma giant has spiked the price of its HIV drug Descovy for providers and pharmacies that deliver care to underserved communities. Gilead claims its HIV drugs do not qualify for its Contract Pharmacy Integrity Initiative.
Real-World Data Study Confirms Safety of Kovaltry in Hemophilia A
A new real-world data (RWD) study has found that switching to Kovaltry did not increase bleeding rates for people living with hemophilia A. The data, published in the European Journal of Haemotology, was came from 318 patients who changed to Kovaltry from FVIII replacement therapies. Patient satisfaction went up after the switch
Report Identifies PCPs Driving Value-Based Care
While nearly 70% of primary care physicians have worked with some form of value-based care, a report by the Chartis Group has zeroed in on characteristics of PCPs that are driving the transition to this payment model. These “Leaders” in value-based care, who are involved in a partial- or full-risk capitation agreement, account for 21% of PCPs, according to the report.
Researchers Team Up to Develop AI-Based Analytics Method to Predict Rare Diseases
A new collaboration between researchers at the University of Florida Health and Penn Medicine seeks to develop new algorithms to predict which patients are at higher risk of developing rare diseases. The researchers will work towards new artificial intelligence (AI) models via their Predictive Analytics via Networked Distributed Algorithms (PANDA) method.
Invitae Teams Up with Astrazeneca in New Rare Cancer Real-World Data Sharing Project
Inivitae and Astrazeneca have announced a new partnership to help advance research for a rare form of cancer, chlangiocarcinoma. As part of the new partnership, Astrazeneca will gain access to Ciitizen, Invitae’s real-world data (RWD) platform. The data comes from the Cholangiocarcinoma Foundation (CCF), a patient advocacy group for people living with this form of bile duct cancer.
Study Finds Elevated Disability Rates Among Transgender Adults
A newly published study in Health Affairs found that transgender adults in the US face significantly higher rates of disability than their peers who are not trans. The study looked at longitudinal data from 2014-2020 collected from the Behavioral Risk Factor Surveillance System. The results find that trans adults are twice as likely to have a disability than their non-trans peers.
Medicaid Still Not Completely Onboard with At-Home Patient Monitoring
At-home patient monitoring is garnering growing attention as a method to improve health outcomes and optimize healthcare spending. However, many states are reticent to let Medicaid reimburse the cost of home monitoring devices. This is, in part, due to a lack of standard operating procedures and mixed data regarding its efficacy and cost-effectiveness.
Clinical Leader Live: Are Decentralized Clinical Trials Cutting Costs at Clinical Sites?
The logistics of the COVID-19 pandemic directly impacted the ability of many pharma and biotech companies to perform research at traditional clinical trial sites. As a result, the use of decentralized clinical trials (DCTs) and hybrid format trials boomed. An episode from earlier this year covers how this has impacted traditional clinical sites and their staff.
FDA Issues Final Guidance on Clinical Trials with Multiple Endpoints
Last week, the US Food and Drug Administration (FDA) released its final guidance on multiple endpoints in clinical trials. The goal of the guidance is to provide standardized strategies for clinical trials with multiple endpoints to avoid ambiguity in study findings that might have bearing on the regulatory approval process and patient outcomes.
Syndicated Report: Health Economics and Outcomes Research (HEOR) Services Market Research Report Forecast 2022 -2029
The health economics and outcomes research (HEOR) market has exploded in recent years, in part due to the ongoing COVID-19 pandemic. Keeping up with this rapidly changing landscape requires deep data-driven insights. In a new syndicated report from Worldwide Market Reports, learn about key industry trends to watch from 2022-2019 to find success in the HEOR field.
TriNetX Announces Launch of its LUCID Trusted Research Environment Evidence Generation Platform
TriNetX has launched its new LUCID – Trusted Research Environment. LUCID allows users to transfer and analyze data within the TriNetX platform. In addition, researchers can use LUCID to transform sources of real-world data (RWD) into usable RWE. LUCID also helps reduce siloing by collecting all resources into one place without having to take data on and off the platform to analyze it.
BeiGene and Ontada Announce New Oncology RWE Collaboration to Advance Patient Access to Therapeutics
The McKesson real-world evidence (RWE) company Ontada and the biotech BeiGene have announced a new collaboration to advance patient access to oncology therapeutics. The team-up will leverage Ontada’s RWE expertise to move towards BeiGene’s goal of bringing 10 drugs to the clinic each year. In addition, the two companies will work together to support the use of RWE in regulatory decision-making in the oncology field.
CVS Health Enters the Clinical Trial Space
Researchers are increasingly eyeing pharmacies as recruitment sites for clinical trials due to their proximity to a wide range of patients. To capitalize on this growing opportunity, CVS Health will begin to host clinical trial recruitment at its numerous sites across the US. In a new article on Applied Clinical Trials, Moe Alsumidaie sits down with Jill Pellegrino, VP of recruitment and real-world evidence (RWE) at CVS Health Clinical Trial Services to talk about the move.
Monkeypox Vaccines May Be Real-World Data’s Time to Shine
Real-world data (RWD) played a pivotal role in the rapid development and regulatory approval process for the current slate of COVID-19 vaccines. Similarly, the development and timely rollout of monkeypox vaccines depends on RWD. For example, RWD is helping inform the move to intradermal injection of Bavarian Nordic’s Jynneos vaccine to help stretch limited supplies.
New Asthma Health Equity Index Helps Identify Needs for Children with Asthma
Researchers presenting at the 2022 AAP National Conference & Exhibition earlier this month put forward a new index that helped identify individual needs of children with asthma. The Asthma Health Equity Index collects and displays data related to social determinants of health (SDoH) to empower health equity efforts. The researchers tested the index by contacting the households of children who had recent emergency department visits for poorly managed asthma.
New Hospital Monitoring Features Added to BioIntelliSense BioButton Wearable
BioIntelliSense’s hospital monitoring wearable BioButton’s feature set will be expanded thanks to the company’s acquisition of AlertWatch. This purchase will combine the massive amount of data collected by the one-time-use device, which tracks patients across a hospital stay, with data pooled from electronic health records (EHR). This empowers approaches to improve care for patients and reduce healthcare costs
Using Community Health Partnerships to Promote Better Health OutcomesUsing Community Health Partnerships to Promote Better Health Outcomes
Social determinants of health (SDoH) play an outsized role in health outcomes. Therefore, targeting these factors through community engagement can play a big role in improving the health of those who traditionally experience health disparities. In a new article, Michael Minor of UnitedHealthcare explains how to foster health equity through partnerships with community-based organizations.
Real-World Evidence Backs Up Safety and Efficacy of Gilead’s Biktarvy for Use as HIV Treatment
Gilead presented new real-world evidence (RWE) at HIV Glasgow 2022 that further supports the use of its drug Biktarvy for the treatment of HIV in people with HIV-related comorbidities. The results, based on real-world data (RWD) from the 2-year follow-up in the BICSTaR study, show that patients taking Biktarvy had a 95-97% rate of achieving viral suppression with limited side effects.
Call for Abstracts: ISPOR 2023
ISPOR has opened abstract submissions for issue panels, workshops, and breakout sessions at its upcoming ISPOR 2023, the leading conference for health economics and outcomes research (HEOR) professionals. ISPOR 2023 takes place in Boston, MA, from May 7th-May 10th, 2023. Abstract submissions for issue panels, workshops, and breakout sessions close by December 15th, 2022. Abstracts for research and case studies open November 2nd and closes January 12th, 20223.
Pfizer Considering 400% COVID-19 Vaccine Price Increase in US
Since their release to the general public last year, the US government has been purchasing and providing COVID-19 vaccines free of charge to patients. With this program set to end soon as public demand for the vaccine draws down, Pfizer is looking to hike its price up by 400% as it enters the commercial market.
Meta-Analysis Confirms Racial Discrimination Worsens Pregnancy Outcomes
A recent meta-analysis in BMJ Global Health concluded that racial discrimination in the US negatively impacts health outcomes for pregnant people. The high maternal mortality rates experienced by Black Americans has been a pressing, but long-sidelined issue in the US. This work helps to pin down racial discrimination as a primary contributor to such health disparities
Effectiveness and Safety of Psoriasis Treatment Validated by Real-World Data Study
A new real-world data (RWD) of guselkumab, Janssen’s therapeutic for plaque psoriasis, confirmed the safety and efficacy that was demonstrated in randomized controlled trials (RCTs). Data from 79 people with moderate to severe plaque psoriasis. The majority of participants had a 90% or higher reduction in the plaque area and intensity index (PASI).
African Health Organizations Commit $100 Million to Expanding Healthcare Workforce
Late last month, a group of health organizations from African countries announced a new $100 million initiative to expand their continent’s healthcare workforces. The group was comprised of membersfrom Malawi, Uganda, Zambia, Sierra Leone, the African Union (AU), and the Africa Centres for Disease Controls (Africa CDC). This is meant to address the health inequity faced by many Africans.
